Updated guidelines for managing patients with antineutrophil cytoplasmic antibody (ANCA)-associated vasculitis (AAV) from the British Society for Rheumatology (BSR) were published recently in ...

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews pediatric neurologist Sithara Ramdas, MD, of Oxford Children’s Hospital in England, on neonatal and juvenile myasthenia gravis.

Physical activity before or during pregnancy was not associated with negative maternal/neonatal outcomes; however, physical activity was not statistically significantly associated with positive ...

A patient with KIT-C -negative systemic mastocytosis (SM) initially presented with severe anemia alongside intraosseous-intramuscular hemorrhage in a case report recently published in Cureus. Atypical ...

In patients with the granulomatosis with polyangiitis (GPA) or microscopic polyangiitis (MPA) subtypes of antineutrophil cytoplasmic antibody (ANCA)-associated vasculitis (AAV) who have kidney ...

Tailored, risk-stratified rituximab regimens including yearly infusions may offer sustained disease control in patients with antineutrophil cytoplasmic antibody (ANCA)-associated vasculitis (AAV), ...

In patients with eosinophilic granulomatosis with polyangiitis (EGPA), a rare form of antineutrophil cytoplasmic antibody (ANCA)-associated vasculitis (AAV), adding cyclophosphamide to glucocorticoid ...

Vosoritide demonstrated sustained long-term efficacy and safety in children with achondroplasia, significantly improving growth outcomes and maintaining high treatment adherence in a real-world ...

There were no significant changes in the levels of a polysialic acid (polySia), a glycoepitope that plays a key role during neuronal development and plasticity, in chronic inflammatory demyelinating ...

Elastase-inhibitory activity (EIA) is associated with chronic obstructive pulmonary disease (COPD) in patients with alpha-1 antitrypsin deficiency (AATD), according to a study published in PLOS One.

Complement inhibition appears to effectively block lesion extension as well as facilitate oligodendrocyte repopulation and myelin repair during acute attacks of neuromyelitis optica spectrum disorder ...

A CRISPR/Cas9-modified Janus kinase 2 (JAK2) V617F model may be useful to develop new therapies against myeloproliferative neoplasms (MPNs), showed a new study published in the International Journal ...